Acorda Announces Safety and Tolerability Data from First Clinical Trial
of Remyelinating Antibody in Multiple Sclerosis

Phase 1 trial results support advancing development of rHIgM22

February 02, 2015 07:11 AM Eastern Time

ARDSLEY, N.Y.--(EON: Enhanced Online News)--Acorda Therapeutics, Inc. (Nasdaq:ACOR) today announced safety and
tolerability data from a Phase 1 clinical trial of rHIgM22, a
remyelinating antibody being studied for the treatment of multiple
sclerosis (MS). The trial, which followed participants for up to six
months after receiving a single dose of rHIgM22, found no dose-limiting
toxicities at any of the five dose levels studied. Based on these data,
the Company intends to advance clinical development of rHIgM22.

“We are currently developing the protocol for
our next Phase 1 clinical trial of rHIgM22. The data from this study
will help inform the design of the next trial, which will enroll people
with MS who are experiencing an active relapse.”

“We’re encouraged by the outcome of this trial, which showed that
rHIgM22 was well-tolerated at all of the dose levels we studied,” said
Anthony Caggiano, M.D., Ph.D., Acorda’s Senior Vice President of
Research and Development. “We are currently developing the protocol for
our next Phase 1 clinical trial of rHIgM22. The data from this study
will help inform the design of the next trial, which will enroll people
with MS who are experiencing an active relapse.”

This was a multi-center, double-blind, randomized, placebo-controlled
study designed to evaluate safety, tolerability, pharmacokinetics, and
immunogenicity of a single dose of rHIgM22 in participants with any type
of MS who were clinically stable for at least three months. All
participants remained on their existing MS treatment regimens, including
disease-modifying therapies.

The first part of the study included five cohorts, with each cohort
receiving a higher dose of rHIgM22 than the previous one. Each cohort
consisted of 10 participants (eight receiving drug, two receiving
placebo), who were followed for three months after receiving a single
dose of study medication. In the second part of the study, 21
treatment-naïve participants were randomized to receive placebo or one
of the two highest doses of rHIgM22 from the first part of the study.
These participants were followed for six months to assess safety and
tolerability. The second part of the study also included several
exploratory clinical, imaging and biomarker measures, which are still
being analyzed. The study was not powered to determine statistical
significance on these measures.

Additional details from the trial will be presented at future medical
meetings.

Safety Findings

Across all of the study groups, 55 participants received one of the five
doses of rHIgM22 and 17 received placebo (no sample size power
calculation was used to determine the number of participants in each
group). There were no dose-limiting toxicities and no serious adverse
events (SAE) in any of the five rHIgM22 dose levels in the study. There
was one SAE of squamous cell carcinoma in a placebo-treated participant.

Multiple sclerosis (MS) is a chronic, usually progressive disease in
which the immune system attacks and degrades the function of nerve
fibers in the brain and spinal cord by destroying myelin (a process
known as demyelination) and eventually the nerve fibers themselves.
Myelin is a fatty layer of membranes that insulates nerves, facilitating
the transmission of electrical impulses through nerve pathways that
control all neurological functions. In people with MS, disruption in
neurological function often leads to impairments in movement,
bowel/bladder function, vision and sexual function.

The cells that make myelin, called oligodendrocytes, can initially
repair myelin damage. As MS progresses, the ability of oligodendrocytes
to repair areas of demyelination is not sufficient to prevent permanent
neurological injury. Currently, there are no therapies that repair or
restore myelin in demyelinating diseases such as MS. If myelin is able
to be repaired, it could restore electrical conduction and may serve to
protect the exposed nerve fiber from further damage.

rHIgM22 is a recombinant human monoclonal antibody identified in the
laboratory of Moses Rodriguez, M.D. at Mayo Clinic. In preclinical
studies, rHIgM22 has been found to protect oligodendrocytes and
stimulate them to repair areas of demyelination. rHIgM22 treatment also
resulted in sustained improvements in motor activity in preclinical
models.

About Acorda Therapeutics

Founded in 1995, Acorda
Therapeutics is a biotechnology company focused on developing
therapies that restore function and improve the lives of people with
neurological disorders. Acorda markets three FDA-approved therapies,
including AMPYRA®
(dalfampridine) Extended Release Tablets, 10 mg, a treatment to improve
walking in patients with multiple sclerosis (MS), as demonstrated by an
increase in walking speed. The Company has one of the leading pipelines
in the industry of novel neurological therapies. Acorda is currently
developing a number of clinical and preclinical stage therapies. This
pipeline addresses a range of disorders including post-stroke walking
deficits, Parkinson’s disease, epilepsy, neuropathic pain, heart
failure, MS and spinal cord injury.

For more information, please visit the Company’s website at: www.acorda.com.

Forward-Looking Statements

This press release includes forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995. All
statements, other than statements of historical facts, regarding
management's expectations, beliefs, goals, plans or prospects should be
considered forward-looking. These statements are subject to risks and
uncertainties that could cause actual results to differ materially,
including the ability to realize the benefits anticipated from the
Civitas transaction and to successfully integrate Civitas' operations
into our operations; our ability to successfully market and sell Ampyra
in the U.S.; third party payers (including governmental agencies) may
not reimburse for the use of Ampyra or our other products at acceptable
rates or at all and may impose restrictive prior authorization
requirements that limit or block prescriptions; the risk of unfavorable
results from future studies of Ampyra or from our other research and
development programs, including CVT-301, Plumiaz, or any other acquired
or in-licensed programs; we may not be able to complete development of,
obtain regulatory approval for, or successfully market CVT-301, Plumiaz,
or any other products under development; we may need to raise additional
funds to finance our expanded operations and may not be able to do so on
acceptable terms; the occurrence of adverse safety events with our
products; delays in obtaining or failure to obtain regulatory approval
of or to successfully market Fampyra outside of the U.S. and our
dependence on our collaboration partner Biogen Idec in connection
therewith; competition; failure to protect our intellectual property, to
defend against the intellectual property claims of others or to obtain
third party intellectual property licenses needed for the
commercialization of our products; and, failure to comply with
regulatory requirements could result in adverse action by regulatory
agencies.

These and other risks are described in greater detail in Acorda
Therapeutics' filings with the Securities and Exchange Commission.
Acorda may not actually achieve the goals or plans described in its
forward-looking statements, and investors should not place undue
reliance on these statements. Forward-looking statements made in this
release are made only as of the date hereof, and Acorda disclaims any
intent or obligation to update any forward-looking statements as a
result of developments occurring after the date of this release.

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